IV C1 Esterase Inhibitor for Hereditary Angioedema
Recruiting in Palo Alto (17 mi)
+12 other locations
Age: Any Age
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: Octapharma
Must not be taking: Androgens, Monoclonal antibodies
Disqualifiers: Pregnancy, B-cell malignancy, Thromboembolic events, others
Pivotal Trial (Near Approval)
Prior Safety Data
Approved in 4 Jurisdictions
Trial Summary
What is the purpose of this trial?Prospective, multicenter, randomized, double-blind, parallel group, placebo- controlled, efficacy and safety phase 3 study of an intravenous human plasma- derived C1 esterase inhibitor (C1-INH) concentrate in participants with congenital C1-INH deficiency for the treatment and pre-procedure prevention of acute hereditary angioedema attacks
Will I have to stop taking my current medications?
The trial requires participants to stop certain medications before starting the study. You must not take specific blood products, hormone therapies, and some pain medications for a period ranging from 7 to 14 days before the trial. Please check with the study team for details on your specific medications.
What data supports the effectiveness of the drug C1 Esterase Inhibitor for treating hereditary angioedema?
Research shows that C1 esterase inhibitor (C1-INH) is effective for managing hereditary angioedema (HAE) attacks, with studies indicating it is the treatment of choice for acute attacks and effective for long-term prevention. Early treatment with C1-INH is thought to improve outcomes, and subcutaneous C1-INH has been proven effective in preventing HAE attacks.
12345Eligibility Criteria
This trial is for individuals with a confirmed diagnosis of Hereditary Angioedema (HAE) types I or II. Participants must have had multiple HAE attacks in recent months and have specific levels of C1-INH activity and C4 antigen. They should agree to follow study procedures, use certain contraceptives if applicable, and be at least 18 years old for phase one or at least 2 years old for phase two.Inclusion Criteria
I am at least 18 years old for the 1st phase or at least 2 years old for the 2nd phase.
I have been diagnosed with HAE type I or II.
I am willing and able to follow all study rules and attend all appointments.
+7 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive either OCTA-C1-INH or placebo injection after the first qualifying attack for the treatment and pre-procedure prevention of acute hereditary angioedema attacks
Single administration per attack
1 visit (in-person) per attack
Follow-up
Participants are monitored for symptom relief and response to treatment after injection
4 hours post-injection
Continuous monitoring for 4 hours
Long-term follow-up
Participants are monitored for long-term safety and effectiveness
4 weeks
Participant Groups
The trial is testing an intravenous treatment called OCTA-C1-INH against a placebo to see if it's effective and safe in treating acute hereditary angioedema attacks. It's a phase 3 study where participants are randomly assigned to receive either the real medication or a placebo in different groups without knowing which they're getting.
2Treatment groups
Experimental Treatment
Placebo Group
Group I: OCTA-C1-INHExperimental Treatment1 Intervention
OCTA-C1-INH injection, 20IU/kg BW after first qualifying attack. Treatment to be administered to blinded as well as open-label subjects.
Group II: PlaceboPlacebo Group1 Intervention
0.1 mL/kg BW 0.9% sodium chloride solution injection after first qualifying attack. Only blinded subjects to receive.
OCTA-C1-INH is already approved in United States, United States, United States, European Union for the following indications:
🇺🇸 Approved in United States as Berinert for:
- Acute abdominal, facial, or laryngeal hereditary angioedema (HAE) attacks
🇺🇸 Approved in United States as Cinryze for:
- Routine prophylaxis against angioedema attacks in adolescent and adult patients with HAE
🇺🇸 Approved in United States as Haegarda for:
- Routine prophylaxis to prevent Hereditary Angioedema (HAE) attacks in patients 6 years of age and older
🇪🇺 Approved in European Union as C1 Esterase Inhibitor (Human) for:
- Hereditary Angioedema (HAE)
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Octapharma Research SiteToledo, OH
Octapharma Research SiteFarmington Hills, MI
Octapharma Research SiteCentennial, CO
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Who Is Running the Clinical Trial?
OctapharmaLead Sponsor
References
Recombinant Human C1-Esterase Inhibitor to Treat Acute Hereditary Angioedema Attacks in Adolescents. [2018]Recombinant human C1-esterase inhibitor (rhC1-INH) is efficacious and well tolerated for managing hereditary angioedema (HAE) attacks in adults. However, there are insufficient data on its efficacy and safety in adolescents.
Effect of time to treatment on response to C1 esterase inhibitor concentrate for hereditary angioedema attacks. [2013]C1 esterase inhibitor (C1-INH) concentrate is well established as effective therapy for hereditary angioedema (HAE). It is thought that treatment of an acute HAE attack with C1-INH as early as possible improves efficacy, but there are limited data from prospective studies supporting this recommendation.
Home therapy with intravenous human C1-inhibitor in children and adolescents with hereditary angioedema. [2017]C1-esterase inhibitor (C1-INH) replacement therapy is the treatment of choice for acute edema attacks in patients with hereditary angioedema (HAE).
Long-Term Outcomes with Subcutaneous C1-Inhibitor Replacement Therapy for Prevention of Hereditary Angioedema Attacks. [2020]For the prevention of attacks of hereditary angioedema (HAE), the efficacy and safety of subcutaneous human C1-esterase inhibitor (C1-INH[SC]; HAEGARDA, CSL Behring) was established in the 16-week Clinical Study for Optimal Management of Preventing Angioedema with Low-Volume Subcutaneous C1-Inhibitor Replacement Therapy (COMPACT).
Elevated D-dimers in attacks of hereditary angioedema are not associated with increased thrombotic risk. [2018]Recommended management of attacks of hereditary angioedema (HAE) due to C1 esterase inhibitor (C1-INH) deficiency (C1-INH-HAE) includes therapy with exogenous C1INH. Thrombotic/thromboembolic events (TEE) have been reported with plasma-derived C1INH, but so far none with recombinant human C1INH (rhC1INH). This phase III, randomized, placebo (saline)-controlled study evaluated the safety of rhC1INH 50 IU/kg for the treatment of acute attacks in 74 patients with C1-INH-HAE.
Safety of C1-esterase inhibitor in acute and prophylactic therapy of hereditary angioedema: findings from the ongoing international Berinert patient registry. [2015]The plasma-derived, pasteurized C1-inhibitor (C1-INH) concentrate, Berinert has a 4-decade history of use in hereditary angioedema (HAE), with a substantial literature base that demonstrates safety and efficacy. Thromboembolic events have rarely been reported with C1-INH products, typically with off-label use or at supratherapeutic doses.
Indirect comparison of intravenous vs. subcutaneous C1-inhibitor placebo-controlled trials for routine prevention of hereditary angioedema attacks. [2022]Label="INTRODUCTION" NlmCategory="BACKGROUND">For prophylaxis of hereditary angioedema (HAE) attacks, replacement therapy with human C1-inhibitor (C1-INH) treatment is approved and available as intravenous [C1-INH(IV)] (Cinryze®) and subcutaneous [C1-INH(SC)] HAEGARDA® preparations. In the absence of a head-to-head comparative study of the two treatment modalities, an indirect comparison of data from 2 independent but similar clinical trials was undertaken.
Angiotensin-converting enzyme inhibitors-induced angioedema treated by C1 esterase inhibitor concentrate (Berinert®): about one case and review of the therapeutic arsenal. [2020]C1 esterase inhibitor (Berinert®) is generally used to treat severe attack of hereditary angioedema. We describe here the case of a patient who presented with a severe angioedema induced by angiotensin-converting enzyme inhibitors (ACEIs) endangering her life. It could be successfully treated with that medicine.
Hereditary angioedema with normal C1 inhibitor: clinical characteristics and treatment response with plasma-derived human C1 inhibitor concentrate (Berinert®) in a French cohort. [2017]Hereditary angioedema (HAE) is a rare genetic disorder characterised by episodes of swelling without urticaria. Berinert® (CSL Behring) is a plasma-derived human C1 inhibitor (C1-INH) concentrate, approved for the treatment of HAE with C1-INH deficiency (C1-INH-HAE), however, it is often used off-label in Europe to treat HAE with normal C1-INH. To report the clinical characteristics of patients with HAE with normal C1-INH (with F12 gene mutation; FXII-HAE) or of unknown origin (U-HAE), and their response to Berinert®. Data from 2007 to 2016 (obtained retrospectively from the French Cohort BeRinert Angiœdème [COBRA] registry of HAE patients with everyday use of Berinert®) were analysed; no control group was included. Diagnostic criteria for FXII-HAE and U-HAE included a normal C1-INH antigenic level and function and refractoriness to high-dose antihistamines. For FXII-HAE, diagnosis also included F12 gene mutation, and U-HAE a positive family history for the disease. To date, 28 patients with FXII-HAE or U-HAE were identified (mean age: 27 years; first angioedema attack at 19.8 years; 85.7% female) with 78 documented Berinert®-treated attacks, the majority occurring in the laryngeal and abdominal regions. Efficacy assessment of Berinert® was available for 38 of 78 documented Berinert®-treated attacks; 22 improved within 60 minutes of treatment initiation, nine within 60-180 minutes, four after 180 minutes, and three showed no improvement. No severe or serious adverse effects were reported. Data to date suggest that Berinert® may be a safe and efficacious treatment option for the majority of HAE patients.
Population pharmacokinetics of plasma-derived C1 esterase inhibitor concentrate used to treat acute hereditary angioedema attacks. [2011]C1 esterase inhibitor (C1-INH) replacement is recommended as a first-line therapy for acute edema attacks in hereditary angioedema (HAE). Only limited pharmacokinetic analyses of the administered C1-INH in plasma are available.
Plasma-derived C1 esterase inhibitor pharmacokinetics and safety in patients with hereditary angioedema. [2023]Over 40 years of use demonstrates that complement 1 esterase inhibitor (C1-INH) concentrate is effective and well tolerated for acute edema attacks and prophylaxis in patients with hereditary angioedema. OCTA-C1-INH is a new stable, virus-inactivated, nanofiltrated concentrate of C1-INH derived from human plasma.